European Medicines Agency: Overview of 2013 For Orphan Drugs
This past week, the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) gave an overview on orphan drugs in 2013. Here are a few highlights :
• In 2013, 13.6 % of the total medicines recommended for marketing authorization by EMA’s Committee for Medicinal Products for Human Use (CHMP) are for the treatment of rare diseases
• # of applications received by EMA for orphan designation increases by more than 22 % since 2009
• # of companies seeking scientific advice offered to sponsors developing orphan medicines during drug development, increases by approximately 32 %, from 2012 to 2013
• Approximately 40 % of applications for orphan designation submitted to the EMA are from small or medium-sized enterprises (SME).
According to Bruno Sepodes, Chair of EMA’s COMP:
“12 years after the European Orphan Regulation came into force, it is undoubtedly serving its purpose, with more and more orphan medicines reaching patients with rare diseases each year. The joint efforts of the EMA committees, including the COMP, the Pediatric Committee, and the CHMP, in providing scientific advice to companies during the development of their medicines, have been a driving force behind this success.”
Since 2012, the EMA, the FDA, and the Japanese regulatory authorities (MHLW and PMDA), are working together to encourage applications for orphan designation to be submitted in parallel to the agencies. For 2013, 50% of applications for orphan designation are submitted in parallel to other regulators. This trend is expected to continue in 2014. To continue the international collaboration of the three regulatory agencies, a joint EMA/FDA/MHLW-PMDA Orphan Medicinal Product Workshop is scheduled for March 10, 2014, at the EMA, in London (UK). There is no registration fee for attending, but registration is required. One can register online. The meeting Agenda is available. This is the third time this workshop is being held and the first time that Japan’s WHLW-PMDA is involved as an organizer. It is an opportunity to encourage “international cooperation for orphan medicines” between three different and unique global regulatory agencies.
Japanese Ministry of Health, Labour, and Welfare (WHLW)
Japanese Pharmaceuticals and Medical Devices Agency (PMDA).